Thursday, July 25, 2013
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Retinal delivery of gene
therapy via intravitreal injection of an engineered adeno-associated viral
Intravitreal injection of
an engineered AAV vector could enable retinal gene therapy. Directed
evolution of AAV libraries identified an AAV variant that, after intravitreal
injection in normal mice and monkeys, entered photoreceptors and other layers
of the outer retina. In mouse models for X-linked retinoschisis or Leber's
congenital amaurosis (LCA), intravitreal injection of the AAV variant expressing
X-linked juvenile 1 (Rs1;
pigment epithelium-specific protein 65 kDa (Rpe65)
decreased signs of disease and increased retinal function compared with
injection of control AAV vectors expressing the respective genes. Future
studies could include testing the AAV variant as a gene therapy vector in
animal models for other congenital retinal diseases.
Published online July 25, 2013
Patented by The
Regents of the University of California; licensed to
an undisclosed entity; available for partnering
Dalkara, D. et al. Sci.
Transl. Med.; published online June 12, 2013;
Contact: David V. Schaffer, University of California, Berkeley,
Contact: John G. Flannery, same affiliation as above
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